Mitochondria-Targeted Delivery of siRNA by Amphiphilic Nanovectors Self-Assembled from a Cationic
Lamiaa M A Ali1, Dan-Dan Su1,2, Rebecca Mucci3
1Institut des Biomolécules Max Mousseron (IBMM), CNRS, ENSCM, Université of Montpellier, Montpellier, France.
Chembiochem : a European Journal of Chemical Biology
|October 7, 2025
Abstract:
The active delivery of therapeutic nucleic acids with subcellular precision is an open challenge. Herein, a small conjugate molecule, combining a cationic [4]helicene moiety with a lipophilic squalene tail, yields amphiphilic nanoassemblies capable of effectively complexing siRNA within the mitochondria. A proof-of-concept in HCT 116 colorectal cancer cells was evidenced by Western blot for the delivery of siRNA silencing the protein expression of the mitochondria-encoded cytochrome c oxidase subunit 1 gene.


